当一位母亲发现，原本活泼好动的儿子在3岁后逐渐变得容易摔倒，爬楼梯越来越吃力；当一位父亲看着孩子从蹒跚学步到需要搀扶行走，心中满是困惑与不安——这可能是杜氏肌营养不良（Duchenne muscular dystrophy，DMD）悄悄降临的信号。

$48.7 billion for NIH, a 1% increase that helps sustain biomedical research critical to rare disease progress. The funding includes positive policy language limiting forward funding of grants and ...

After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments ...

Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.

KYOTO--Utilizing genome editing technology, a team of researchers has developed a much more effective treatment for a certain ...

Disability advocate TaLisha Gryzb is a mother of four living with muscular dystrophy who educates viewers about her ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

A GoFundMe has been launched for Liam Cusack, an Irish American journalist who is battling Multiple Myeloma, a deadly blood ...